Biomarker-derived fast-and-frugal decision tree for preemption of veno-occlusive disease/sinusoidal obstructive syndrome

Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative therapy for blood disorders. However, the ef fi cacy of this procedure has been impeded by early endothelial dysfunction, which can lead to a severe and potentially lethal complication called veno-occlusive disease (VOD)/sinusoidal obstructive syndrome (SOS). Despite less aggressive conditioning regimens leading to a decrease in incidence in recent years, VOD/SOS that evolves into multiorgan dysfunction syndrome has a high mortality rate. 1 As highlighted by the Pediatric Acute Lung Injury and Sepsis Investigators and Pediatric Blood and Marrow Transplantation Consortium Joint Working Committee consensus, there is high variability in pediatric management of VOD/SOS, which may contribute to the increased morbidity and mortality. 2 De fi brotide received Food and Drug Administration approval for VOD/SOS treatment, 3-5 and its safety pro fi le is excellent. 6 In a randomized trial, its prophylactic administration in a pediatric population resulted in decreased VOD/SOS incidence from 20% to 12%. 7 This was not con fi rmed in a trial including adults and children. 8 The observed difference could be due to different risk pro fi les between 2 populations indicating the need for developing individualized care for the management of VOD/SOS. In a prospective cohort of 80 pediatric HCT, a model based on 3 biomarkers (L-fi colin, hyaluronic acid, and stimulation 2) accurately strati fi ed these patients in high (>30%) vs low (<5%) risk groups for developing VOD/SOS. 9 However, improved risk strati fi cation for VOD/